I’m a sucker for sick kids, it just seems so unfair that a child should have to suffer through something that I know would crush me if I had to go through it. At the beginning of last year I came across Courtney Roth’s blog, EBing A Mommy, about her son Tripp’s battle with junctional Epidermolysis bullosa. Epidermolysis bullosa is a devastating skin disease that causes the skin to blister – inside and out – at the slightest touch or friction, and junctional EB is the worst of its kind. Tripp had just turned two when I started reading, an amazing feat as doctors didn’t suspect he’d make it past the age of 1. Courtney and Tripp’s story was heart wrenching and uplifting, they were amazing and strong in the face of the greatest adversity.

Tripp passed away on Saturday, January 14^th. I bawled when reading Courtney’s blog entry. This little boy I had never met had a special place in my heart, in thousands of hearts, he was just an amazing little guy.

S. 606/HR 3059, the Creating Hope Act, is set to help children with rare diseases. Here is the official summary:

9/23/2011–Introduced.Creating Hope Act of 2011 – Amends the Federal Food, Drug, and Cosmetic Act to
revise the priority review voucher program for tropical diseases to:

1. include rare pediatric diseases;
2. allow unlimited transfers of vouchers under the program;
3. require a sponsor intending to use a voucher to notify the Secretary of Health and Human Services (HHS) at least 90 days (currently, 365 days) before submission of the application.

Revises provisions regarding the priority review user fee, including to make it payable upon notification of the Secretary of intent to use the voucher. Authorizes the Secretary, upon request of the manufacturer or sponsor of a new drug, to designate that a new drug is:

1. for a rare pediatric disease;
2. an eligible treatment.

Requires the Secretary to deem a rare pediatric disease product application to be incomplete if it does not contain a description of the sponsor’s plan to market the product in the United States. Authorizes the Secretary to refuse to issue a priority review voucher upon the approval of a rare pediatric disease application if the Secretary finds that the sponsor lacks a good faith intention to market the product in the United States. Directs the Secretary to issue a guidance document setting forth the evidentiary support necessary to demonstrate such a good faith intention. Requires sponsors of applications under the priority review voucher program to report to the Secretary within five years after the approval of the application on the demand and distribution of the product within the United States.

Simply put, this bill will help to encourage companies to put time and money into researching rare diseases that normally don’t receive such care. It’s a pretty big deal for kids like Tripp who suffer from diseases that most of us know nothing about¹.

You can follow this link and put in your contact information to automatically look up your local representatives and send them an email asking them to support the Creating Hope Act. I was able to quickly and easily send a message to Senator Jim Webb (D-VA), Senator Mike Warner (D-VA), and Representative Morgan Griffith (R-VA-09). I’m asking everyone I know to do the same. Speak out for those who can’t.

1. I had never even heard of EB before stumbling onto Courtney’s blog. [↩]

Ramblings